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Showing 1 to 4 of 4 for “"Niemann-Pick disease type C"”.

  1. SYNTHESIS, CHARACTERIZATION, IN VITRO EVALUTION, AND PRECLINICAL PROFILING OF β-CYCLODEXTRIN POLYROTAXANE FAMILIES FOR USE AS POTENTIAL NIEMANN-PICK TYPE C THERAPEUTICS

    Niemann-Pick Disease Type C (NPC) is a rare, autosomal recessive genetic disorder featuring a loss of proteins responsible for unesterified cholesterol (UC) trafficking through the late endosomes/lysosomes (LE/LY) of every cell of the body. Disruption of this pathway leads to abnormal accumulation …

    purdue-thes Repository record for SYNTHESIS, CHARACTERIZATION, IN VITRO EVALUTION, AND PRECLINICAL PROFILING OF β-CYCLODEXTRIN POLYROTAXANE FAMILIES FOR USE AS POTENTIAL NIEMANN-PICK TYPE C THERAPEUTICS (opens in a new tab)

  2. Graphene Brain on a Chip Platform for the Study of Neurodegeneration

    … This network is disrupted in neurodegenerative diseases---such as Parkinson's disease (PD), and there are limited tools available to study the molecular mechanisms behind the degradation of the neuronal network. This indicates a gap in the understanding of how neurodegenerative diseases and PD …

    cambridge Repository record for Graphene Brain on a Chip Platform for the Study of Neurodegeneration (opens in a new tab)

  3. Sterol Binding to Niemann-Pick Type C1 Disease Protein (NPC1) - Implications for its Function in Cholesterol Transport

    … in animal cells remains obscure. Studies on Niemann-Pick type C disease (NPC) defined the requirement of at least two proteins involved in the transport of lipoprotein-derived cholesterol from lysosomes. Both proteins are located in the lysosomes and mutations in either NPC1, a membrane bound …

    utswmed Repository record for Sterol Binding to Niemann-Pick Type C1 Disease Protein (NPC1) - Implications for its Function in Cholesterol Transport (opens in a new tab)

  4. Evaluating the Mechanisms of 2-Hydroxypropyl-β-Cyclodextrin and Liver X Receptor Agonists as Potential Therapies for Niemann-Pick Type C Disease

    … cholesterol are tightly regulated. In some rare disease states, such as in Niemann-Pick Type C, cholesterol balance is lost leading to detrimental effects. In Niemann-Pick Type C, mutations in either of the cholesterol trafficking proteins, NPC1 or NPC2, lead to the entrapment of unesterified …

    utswmed Repository record for Evaluating the Mechanisms of 2-Hydroxypropyl-β-Cyclodextrin and Liver X Receptor Agonists as Potential Therapies for Niemann-Pick Type C Disease (opens in a new tab)